Pipeline - Affinia Therapeutics

Purposeful science – Lasting patient impact

We are building a pipeline of rationally designed gene therapy product candidates, pioneering a shift to treat not only rare but also prevalent diseases.

Our initial focus is on diseases of the heart, where we foresee a transformational change in the treatment paradigm similar to what has happened in the treatment of cancers. This is because advances in science are identifying new genes associated with diseases of the heart, making a compelling case for genetic medicines. The heart represents an organ where targeted delivery of the therapeutic genetic payload is currently inadequate. Our ART platform provides us with an advantage to address these limitations and develop potentially first-in-class or best-in-class product candidates that may be safer and more effective.

Genetic medicines for the heart

We have applied ART to rationally design a new class of capsids with improved targeting of the heart muscle. In animal studies, our novel capsids such as ATC-187 administered as a simple intravenous (IV) infusion have been shown to reach almost all heart cells with homogeneous distribution. In contrast, conventional capsids such as AAV9 or AAVrh74 reached less than 20% of cells at the same dose with a sporadic, patchy distribution pattern. The doses we studied are 5-10 times lower than what is currently being tested in gene therapy trials for cardiac diseases. Reaching the predominant number of cardiomyocytes in a homogenous manner has been a long-standing limitation in the treatment of diseases of the heart such as BAG3-associated dilated cardiomyopathy (DCM). We believe we’ve solved this limitation. The lower doses used for Affinia’s capsids have the potential to also reduce dose-related safety issues such as liver injury, complement activation, and dorsal root ganglia injury and also allow a less intense regimen of immunosuppressants than that typically used with conventional capsids. We’ve presented our findings at multiple scientific conferences. To see this breakthrough science, please click here.

Research efforts in other heart diseases

We are testing a number of additional product candidates in preclinical studies to evaluate safety and effectiveness in other devastating diseases affecting the heart.

We’ve presented our findings at a scientific conference. To see this breakthrough science, please click here.

View Breakthrough Science

Genetic medicines for the nervous system

We have applied ART to rationally design a new class of capsids that target the nervous system by crossing the blood-brain-barrier (BBB), a protective system that generally prevents capsids from reaching the brain. In animal studies, our novel capsids administered via a simple intravenous infusion have been shown to cross the BBB and reach the majority of neurons, which are the main cell types of interest in many CNS diseases. In contrast, conventional capsids such as AAV9 reached less than 5% of neurons at the same dose. Crossing the BBB has been considered a key barrier to the treatment of diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and Huntington’s disease. We’ve presented our findings at scientific conferences. To see our breakthrough science, please click here.

Our Pipeline

Purposeful science – Lasting patient impact

Genetic medicines for the heart

Research efforts in other heart diseases

Genetic medicines for the nervous system

Explore the future of gene therapy

Learn why Affinia Therapeutics is the future of gene therapy

Read about the latest progress at Affinia Therapeutics

Contact us

Our address

Our Company

Our Science

Careers

Contact Info

Office Address